Axovant Sciences (NASDAQ: AXON), a clinical-stage company developing innovative gene therapies for neurological conditions, today announced feedback from a face-to-face pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding AXO-Lenti-PD for patients with Parkinson's disease. Based on the discussion at the meeting, the totality of data collected on the initial vector construct, ProSavin, including over six years of phase 1/2 clinical data and IND-enabling preclinical data, may be supportive of the planned development program for AXO-Lenti-PD. 

The phase 2 clinical trial of AXO-Lenti-PD (NCT03720418), now called SUNRISE-PD, was initiated in the U.K. in the fourth quarter of 2018. The SUNRISE-PD study is advancing as planned with dosing of the second patient in November 2018. To date, both patients tolerated the surgical procedure well and were discharged home with no serious adverse events observed. Axovant expects to announce data from the first two patients in March 2019.

During the meeting discussion and subsequent written meeting minutes, Axovant received feedback from the FDA on several key features of the AXO-Lenti-PD development program:

The target patient population will be adult patients with Parkinson's disease who are refractory to additional medical management due to motor complications

The ongoing SUNRISE-PD clinical study of AXO-Lenti-PD constitutes an early-phase, exploratory trial that may support a future marketing application if safety and efficacy data are meaningful

The primary efficacy measure of the randomized, sham-controlled portion of the phase 2 study will be assessed at 12 months using data from Hauser patient diaries

Additional secondary efficacy data will be collected on the UPDRS Part III "OFF" score, a motor function assessment completed by clinicians after oral levodopa has been washed out

The proposed current manufacturing process and quality control testing is adequate for the clinical program

FDA agreed in principle with the proposed approach to demonstrate compatibility between the current manufacturing process and the planned serum-free, suspension manufacturing process that will be used to support scale-up and commercialization.

In addition, Axovant was encouraged to return for an End-of-Phase 2 meeting after completion of the ongoing SUNRISE-PD study, during which the available data generated in the study will be discussed in the context of a pivotal program design.

"We are pleased with the feedback received at our meeting with the FDA, which reaffirmed our strategy to view the ongoing SUNRISE-PD clinical study of AXO-Lenti-PD as a continuation of the previous ProSavin program, and part of a single development program," said Gavin Corcoran, M.D., executive vice president of research and development for Axovant. "We believe we are well-positioned to continue to execute on the clinical trial as designed, manufacture drug product at scale, and activate clinical trial sites in the U.S. during the randomized, sham-controlled portion of our SUNRISE-PD study."